Johan Rockberg honoured by the Novo Nordisk Foundation
KTH researcher Johan Rockberg has been awarded the title of “Distinguished Innovator” by the Novo Nordisk Foundation along with 11 million Swedish kronor in research funding. The money will go towards further development of the gene therapy platform developed by Rockberg’s research team to enable targeted treatment of kidney diseases.
“Chronic kidney disease affects more than 800 million people, and a third of cases are caused by DNA mutations that could be cured with an effective gene therapy,” Rockberg says.
The KTH-developed method enables the targeted delivery of DNA via tailor-made viruses, known as viral vectors, which find their way to the right location in the body. The researchers intend to use these viruses to deliver the DNA the patient needs directly to the kidney cells to restore kidney function.
At present, Rockberg’s group has a functional general platform in the preclinical phase. They have engineered the virus’s surface proteins so that they recognise specific receptors located on the cells they wish to target.
“In animal models, we see that our vectors find their way to the desired organs to a significantly greater extent than the vectors used today, whilst avoiding uptake in organs that are not relevant,” Rockberg says.
Modular targeting
The viral vectors currently used in gene therapy largely end up in the liver. This is a fundamental problem when the target organ is the kidney, which is itself difficult to reach with targeted drug delivery. The researchers tackled this challenge from a protein engineering perspective by attaching small, engineered binding proteins to the virus’s capsid (the outer, protective protein shell) and directing them towards selected receptors on the kidney cells.
“This makes the targeting modular; if we change the binding proteins, we change the type of cell targeted, without having to rebuild the entire vector from scratch,” Rockberg says.
Now, the researchers focus on two things: further developing and evaluating kidney-specific viruses and measuring their effectiveness in restoring kidney function. There are still several years of preclinical work ahead before a first study in humans can be considered. The funding and award from the Novo Nordisk Foundation are therefore very welcome.
”This is the result of many years of methodological development, which can now be taken forward to practical applications. Drug development requires a long-term perspective, and this type of funding enables us to take a significant step towards future applications for kidney diseases in collaboration with clinical researchers,” Rockberg says.
Jon Lindhe